A Clinical Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788

A Phase III Study in Patients With Chronic Idiopathic Thrombocytopenic Purpura in R788

The purpose of this study is to investigate the efficacy, safety and pharmacokinetics of R788 compared with placebo, and to investigate the safety and efficacy of long term dosing of R788 in patients with chronic idiopathic thrombocytopenic purpura.

Study Overview

• Status: Completed
• Conditions: Idiopathic Thrombocytopenic Purpura
• Intervention / Treatment: Drug: Placebo; Drug: R788

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Multiple Locations, Japan
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Japanese patients
• Patients diagnosed with idiopathic thrombocytopenic purpura at least 6 months before acquisition of consent
• Patients with a platelet count averages <30000/μL during screening period. Each platelet count should not exceed 35000/μL.
• Patients who have used and failed or who were intolerant at least 1 typical regimen for the treatment of ITP before informed consent (with or without splenectomy)

Exclusion Criteria:

• Patients with thrombocytopenia associated with other disease
• Patients with autoimmune hemolytic anemia
• Patients with poorly controlled hypertension
• Patients with a history or active coagulopathy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: R788; Patients are administered R788 for 24 weeks (double-blind period), followed by R788 for up to 52 weeks (open-label period). Patients who have completed open-label period and meet the criteria are eligible to continue R788 treatment over a 3 year period (extension - period).	Drug: R788; Oral administration
Placebo Comparator: Placebo; Patients are administered Placebo for 24 weeks (double-blind period), followed by R788 for up to 28 weeks (open-label period). Patients who have completed open-label period and meet the criteria are eligible to continue R788 treatment over a 3 year period (extension - period).	Drug: Placebo; Oral administration; Drug: R788; Oral administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of patients with stable platelet response	Percentage of patients with a stable platelet response by Week 24 defined as a platelet count of ≥ 50000/μL on at least 4 of 6 visits between Week 14 to Week 24	24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of patients with overall response	Percentage of patients with a platelet count ≥50000/μL on at least 1 of 6 visits from Week 2 to Week 12	12 weeks
Duration of maintained platelet count	Duration of maintained platelet count since first achievement of a platelet count ≥50000/μL after administration of the study drug	52 weeks
Percentage of patients with a platelet count ≥50000/μL	Percentage of patients with a platelet count ≥50000/μL at the specified evaluation time point	Every 2 weeks up to Week 24, every 4 weeks up to Week 52 and every 8 weeks up to 3 years
Percentage of patients with a platelet count increase ≥20000/μL above baseline and ≥30000/μL	Percentage of patients with a platelet count increase ≥20000/μL above baseline and ≥30000/μL at the specified evaluation time point	Every 2 weeks up to Week 24, every 4 weeks up to Week 52 and every 8 weeks up to 3 years

Collaborators and Investigators

• Sponsor: Kissei Pharmaceutical Co., Ltd.
• Investigators: Study Director: Yoshitaka Shimizu, Kissei Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 24, 2019
• Primary Completion (Actual): December 21, 2021
• Study Completion (Actual): September 25, 2023

Study Registration Dates

• First Submitted: October 15, 2019
• First Submitted That Met QC Criteria: October 16, 2019
• First Posted (Actual): October 18, 2019

Study Record Updates

• Last Update Posted (Estimated): December 21, 2023
• Last Update Submitted That Met QC Criteria: December 15, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: R788-1301

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No